PUBLICATIONS

Designs of the clinical trials aiming at evaluating cell and gene therapy products: A critical appraisal from a literature review
Author(s): Lucie Biard, Vincent Lévy, Sylvie Chevret - on behalf of the JOIN4ATMP consortium
doi: https://doi.org/10.1016/j.omta.2025.201651
This is a systematic literature review of ATMP trials from 2022 to 2024 examining the methodological and statistical aspects of current practice in clinical evaluation of ATMPs and including 276 studies including both cell and gene therapies. “To our knowledge, this is the first literature review that comprehensively examined current practice in clinical evaluation of ATMPs, regardless of regulatory approvals,” the authors state.

Unlocking the full potential of rare disease drug development: exploring the not-for-profit sector’s contributions to drug development and access
Author(s): Stefano Vavassori, Sean Russell, Celeste Scotti, Stefano Benvenuti
Frontiers in Pharmacology
doi: 10.3389/fphar.2024.1441807